Global Patent Index - EP 1927660 A3

EP 1927660 A3 20080611 - Methods for delivering DNA to muscle cells using recombinant adeno-associated virus virions

Title (en)

Methods for delivering DNA to muscle cells using recombinant adeno-associated virus virions

Title (de)

Verfahren zur Abgabe von DNA an Muskelzellen mithilfe rekombinanter adeno-assoziierter Virus-Virionen

Title (fr)

Procédé pour fournir l'ADN à des cellules musculaires utilisant des virions de virus adéno associés recombinants

Publication

EP 1927660 A3 20080611 (EN)

Application

EP 07014403 A 19970117

Priority

  • EP 97904823 A 19970117
  • US 58835596 A 19960118

Abstract (en)

[origin: US5858351A] The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue is disclosed. The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of the delivered gene and for in vivo secretion of the therapeutic protein from transduced muscle cells such that systemic delivery is achieved.

IPC 8 full level

C12N 15/16 (2006.01); A61K 31/70 (2006.01); A61K 38/18 (2006.01); A61K 38/47 (2006.01); A61K 48/00 (2006.01); C07K 14/505 (2006.01); C12N 5/08 (2006.01); C12N 5/10 (2006.01); C12N 7/01 (2006.01); C12N 9/24 (2006.01); C12N 15/86 (2006.01); C12N 15/864 (2006.01); A61K 38/00 (2006.01)

CPC (source: EP US)

A61K 38/1816 (2013.01 - EP US); A61K 38/47 (2013.01 - EP US); A61K 48/00 (2013.01 - EP US); A61P 43/00 (2017.12 - EP); C07K 14/505 (2013.01 - EP US); C12N 9/2471 (2013.01 - EP US); C12N 15/86 (2013.01 - EP US); C12Y 302/0102 (2013.01 - EP US); C12Y 302/01023 (2013.01 - EP US); A01K 2217/05 (2013.01 - EP US); C12N 2750/14143 (2013.01 - EP US); C12N 2830/42 (2013.01 - EP US); C12N 2840/44 (2013.01 - EP US)

Citation (search report)

  • [X] WO 9528493 A1 19951026 - UNIV ROCKEFELLER [US], et al
  • [X] WO 9534670 A2 19951221 - CHILDRENS HOSPITAL INC [US]
  • [X] WO 9520671 A1 19950803 - RHONE POULENC RORER SA [FR], et al
  • [X] WO 9600006 A1 19960104 - UNIV PITTSBURGH [US]
  • [PX] WO 9614061 A1 19960517 - CELL GENESYS INC [US]
  • [E] WO 9712050 A1 19970403 - UNIV ROCKEFELLER [US], et al
  • [X] KAPLITT M G ET AL: "LONG-TERM GENE EXPRESSION AND PHENOTYPIC CORRECTION USING ADENO-ASSOCIATED VIRUS VECTORS IN THE MAMMALIAN BRAIN", NATURE GENETICS, NEW YORK, NY, US, vol. 8, 1 October 1994 (1994-10-01), pages 148 - 154, XP002005767, ISSN: 1061-4036
  • [A] TRIPATHY S K ET AL: "STABLE DELIVERY OF PHYSIOLOGIC LEVELS OF RECOMBINANT ERYTHROPOIETINTO THE SYSTEMIC CIRCULATION BY INTRAMUSCULAR INJECTION OF REPLICATION-DEFECTIVE ADENOVIRUS", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA, NATIONAL ACADEMY OF SCIENCE, WASHINGTON, DC, US, vol. 91, November 1994 (1994-11-01), pages 11557 - 11561, XP002030822, ISSN: 0027-8424
  • [A] PODSAKOFF G ET AL: "LONG-TERM IN VIVO GENE EXPRESSION IN MUSCLE USING AAV VECTORS", BLOOD, W.B. SAUNDERS, PHILADELPHIA, VA, US, vol. 86, no. 10, SUPPL 1, 15 November 1995 (1995-11-15), pages 1004A, XP000673499, ISSN: 0006-4971
  • [A] KOTIN R M: "PROSPECTS FOR THE USE OF ADENO-ASSOCIATED VIRUS AS A VECTOR FOR HUMAN GENE THERAPY", HUMAN GENE THERAPY, MARY ANN LIEBERT, NEW YORK ,NY, US, vol. 5, 1994, pages 793 - 801, XP000651491, ISSN: 1043-0342
  • [PX] PODSAKOFF, G.M., ET AL .: "AAV VECTOR-MEDIATED GENE DELIVERY TO SKELETAL MUSCLE IN VIVO RESULTS IN SUSTAINED LEVELS OF SYSTEMIC ERYTHROPOIETIN", BLOOD, vol. 88, no. 10, November 1996 (1996-11-01), SUPPL1 (PART 1 OF 2), pages 1066, XP000673500
  • [PX] KESSLER, P.D., ET AL .: "GENE DELIVERY TO SKELETAL MUSCLE RESULTS IN SUSTAINED EXPRESSION AND SYSTEMIC DELIVERY OF A THERAPEUTIC PROTEIN", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE USA, vol. 93, November 1996 (1996-11-01), pages 14082 - 14087, XP002032009

Designated contracting state (EPC)

AT BE CH DE DK ES FI FR GB GR IE IT LI LU MC NL PT SE

DOCDB simple family (publication)

US 5858351 A 19990112; EP 1927660 A2 20080604; EP 1927660 A3 20080611; US 2001001661 A1 20010524; US 2002147172 A1 20021010; US 2008199442 A1 20080821; US 6211163 B1 20010403; US 6391858 B2 20020521; US 7238674 B2 20070703

DOCDB simple family (application)

US 58835596 A 19960118; EP 07014403 A 19970117; US 30904299 A 19990510; US 75573401 A 20010104; US 82127107 A 20070622; US 9245402 A 20020305